#ScienceSaturday: May 2, 2020

#ScienceSaturday posts share relevant and exciting scientific news with the KAND community. This project is a collaboration between KIF1A.ORG’s Research Engagement Team Leader Alejandro Doval, President Kathryn Atchley and Science Communication Director Dr. Dominique Lessard. Send news suggestions to our team at impact@kif1a.org.

KIF1A-Related Research: From the Archives

Instead of sharing a recent piece of KIF1A-related research this week, we’re highlighting an influential piece of KIF1A literature from the past. What better way to kick off this series than to feature the article that introduced KIF1A to the research world! In this article we first learned about KIF1A’s essential role in axonal transport within our neurons. We also learned of many types of cargo transported by KIF1A, further highlighting the role of KIF1A in our brains. Since this publication, we have learned a lot about how many KIF1A proteins are needed to transport cargo. It was first proposed that KIF1A functions as a monomer (one KIF1A protein). Based on 25 years of research since this article, we now know that KIF1A is most efficient at transporting cargo as a dimer (two KIF1A proteins working as a functional unit).

Rare Disease News

RNA therapeutics on the rise

With our recently announced collaboration with Ionis, we thought we’d share this short review about the historical rise of RNA therapeutics. This review covers three areas: trends in RNA therapeutic investment, RNA therapeutics pipeline, and the success of RNA therapeutics in treating rare diseases.

RNA therapeutics have at last reached the point of profitability. Multiple oligonucleotide drugs are approved and a dozen more are in phase III trials, primarily for genetically well-defined rare diseases. The current bolus of investment in RNA therapeutics is likely to lead to further clinical success across multiple modalities and disease areas.

One hallmark of neurodegenerative disease is the breakdown, or degeneration, of the axons in our neurons. Unlike other areas in our bodies, damage to axons is considered to be permanent and can lead to an improperly functioning nervous system. This article covers a recent advancement in our understanding of how damaged axons may be repaired. One protein in particular, Lin28, is an enticing option for helping fix axonal damage. Lin28 is a protein that binds to RNA, a type of genetic material in our bodies. Upon binding to RNA, Lin28 can help promote cell growth and repair. Researchers have found that Lin28 is “a major regulator of axon regeneration” in a spinal cord or optic nerve injury mouse model. While this study is mainly focused on Lin28’s role in acute axonal injury, we can use this information to shape our understanding of axonal breakdown resulting from chronic neurodegeneration. Click the image below to read more!

Designed a new family of molecules with high affinity to join altered receptors in neurodegenerative diseases

When looking for new small-molecule drugs, researchers are often looking for different types of receptors to target. If we think about a small-molecule drug as being a “key,” we can think of the receptor as being the appropriate “lock.” Finding the right molecule for the right receptor is like finding the perfect match between a lock and a key. This article discusses a new group of small molecules that target a specific type of receptor called imidazoline receptors. Imidazoline receptors are altered in the brains of Huntington’s, Parkinson’s, and Alzheimer’s disease patients, making them a potential therapeutic target for neurodegenerative diseases.